Is the government’s new industrial strategy a new chance for medical research charities to lead the way?

Does the publication of the government’s Industrial Strategy White Paper provide an unexpected lifeline for the millions of people affected by sight loss? Fight for Sight, the eye research charity, believes so.

Michele Acton, the charity’s CEO, says: “The White Paper presents an opportunity to transform lives by being bold and radical in the approach to innovation and by building on world class research being undertaken in the UK.”

Sight loss is something which affects people of all ages. The most common cause of sight loss in the UK is age-related macular degeneration (AMD) for which there is currently no cure. There are over 600,000 people over the age of 60 living with AMD, a figure that is set to double by 2050. Over 25,000 children in the UK are visually impaired. Total eye appointments accounted for over 8 million NHS outpatient appointments in 2016/17, the second highest of any speciality.

Despite the high number of people affected and the fact that surveys show that loss of sight is one of the most feared health conditions, sight loss remains a neglected disease when it comes to investing in research. Delivering the ambition set out in the White Paper could make a dramatic difference in finding solutions for one of the UK’s major healthcare challenges.

There are a number of strands of the White Paper that are inextricably linked to the challenge of ending sight loss. The 5 foundations of productivity mirror the conditions necessary for Fight for Sight funding of world class eye research to deliver treatments to patients. The UK medical research sector needs good ideas, skilled people, investment in infrastructure, a vibrant economic environment to nurture new businesses and great places in which to develop the research base. As science, medicine and technology come closer together to deliver solutions the emphasis on grand challenges around the development of artificial intelligence (AI) and the Data Economy and an Ageing Society are ever more important. The life sciences sector deal, through the Health Advanced Research Programme (HARP) and other initiatives, presents the opportunity for the public, private and charitable sectors to work collaboratively to innovate and pioneer treatments that can transform lives.

The eye research sector is well placed to help deliver the ambitions set out in the white paper:1. World-class skills, knowledge, expertise and facilities. The UK has some of the world’s leading ophthalmologists. According to the 2017 Centre for World University Rankings (CWUR) by subject, UCL Institute of Ophthalmology is the best place in the world to study ophthalmology, ahead of Harvard and John Hopkins University. But it’s not just at UCL and other universities in London. Our knowledge and expertise is located in universities and hospitals across the UK;

2. The cutting edge of new science. Research into eye diseases and conditions is leading the new frontier of research in the 21st century. Gene and stem cell therapy developments have led to clinical trials not thought possible a decade ago. Smart phones have been adapted to perform full eye examinations. Artificial intelligence is being developed to analyse eye scans to detect disease early.

But there is so much more that needs to be done to unlock potential. No single sector can achieve this alone and charities have a vital role to play in funding early stage research that marks the start of the innovation pipeline. Few funders are focussed on a social return and Fight for Sight sees its role as catalysing innovation, de-risking research with the aim of leveraging further funding from Government and the private sector.

At Fight for Sight, they know this strategy works. The progression of research to find a treatment for choroideremia provides a clear demonstration. Choroideremia is an inherited eye disease that blinds men by the time they are in their forties and for which there is no treatment.

In partnership with patients, Fight for Sight committed £300,000 from 2008 to 2011 to fund vital basic / pre-clinical work at Imperial College London. This enabled Imperial to collaborate with Oxford University to receive a £1.1m grant from the Department of Health and Wellcome for a Phase I gene therapy trial – the world’s first trial of a treatment for choroideremia. After publication of the results of this trial the Department of Health awarded a £1.6m grant for a Phase II trial and Nightstar was spun out of Oxford University with £17m of funding from Syncona, an evergreen fund. Nightstar has subsequently raised over £110m with the aim of taking gene therapy treatments for choroideremia and other inherited eye diseases through to the clinic. With an Initial Public Offering on NASDAQ in September 2017, Nightstar now has a market capitalisation of over $520 million (as of 28 November 2017).

This kind of pipeline progression highlights a number of key factors pertinent to the success of the Industrial Strategy: • Charity and funding from patients provided crucial high risk funding at a stage that is unattractive to most funders;• The eye research sector provided the ideas and people with the necessary skills who were able to progress research in appropriate facilities;• Government provided further capital for early stage trials; and • Evergreen funding provided capital to a new business ahead of venture capital investment.

This is just one example. There are many eye diseases and conditions that we still do not know how to prevent or treat and ending sight loss remains one of the biggest health challenges that faces the UK. The White Paper sets out a pathway, but success will not happen by chance. It will require strong and bold leadership and all sectors working together. Failure to do so will result in a significant missed opportunity that will affect the lives of so many.

The voluntary sector is committed to playing its role. Their interests are aligned with the Government’s ambition of not only improving the lives of patients but also driving economic growth by making the UK an attractive location for life sciences research and development. Fight for Sight believes: “We now need to make it happen.”

Notes to editors:Fight for Sight is the leading UK charity dedicated to funding pioneering research to prevent sight loss and treat eye disease.

Fight for Sight’s overall research commitments amount to £8m for over 159 research projects at 44 different universities and hospitals across the UK.

Fight for Sight is funding research to stop sight loss caused by both common and rare eye diseases and conditions. These include age-related macular degeneration, glaucoma, diabetic retinopathy, inherited eye diseases and the causes of childhood sight loss. Research that has been funded by Fight for Sight has resulted in:• the identification of new genes responsible for glaucoma, retinitis pigmentosa, keratoconus and other corneal disorders, and Nance-Horan syndrome• the world’s first clinical trial of a treatment (a gene therapy) for choroideremia, an inherited condition that causes blindness in men• the design of a new test that can detect the early stages of sight loss in age-related macular degeneration

Fight for Sight social media:https://www.facebook.com/fightforsightuk or https://twitter.com/FightforsightUK